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A cross‐sectional natural history study of aspartylglucosaminuria

Aspartylglucosaminuria (AGU) is a rare lysosomal storage disorder that causes stagnation of development in adolescence and neurodegeneration in early adulthood. Precision therapies, including gene transfer therapy, are in development with a goal of taking advantage of the slow clinical course. Under...

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Detalles Bibliográficos
Autores principales:	Goodspeed, Kimberly, Horton, Daniel, Lowden, Andrea, Sguigna, Peter V., Booth, Timothy, Wang, Zhiyue J., Edgar, Veronica Bordes
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley & Sons, Inc. 2022
Materias:	Research Reports
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9458605/ https://www.ncbi.nlm.nih.gov/pubmed/36101820 http://dx.doi.org/10.1002/jmd2.12294

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